Leveraging Delivery Approaches to Translate Oligonucleotide-Based Therapies for Different Indications
Time: 9:05 am
day: Pre-Conference Workshop Day
Details:
The success of oligonucleotide therapeutics hinges not only on target selection and chemistry but also on delivery strategies that can unlock extrahepatic targets and expand the reach of these modalities. While conjugate-based approaches have driven remarkable progress in hepatocyte delivery, new indications, from neuromuscular diseases to oncology, demand innovative delivery solutions to overcome biological barriers, ensure tissue specificity, and maintain safety.
This interactive workshop will equip you with a deep understanding and overview of delivery innovation, from conjugation technologies and ligand selection to lipid nanoparticles, brain shuttles, and beyond. Through real-world case studies, collaborative problem-solving, and expert-led discussion, you’ll leave with actionable strategies to optimize delivery for different tissue targets, reduce off-target risks, and accelerate your program toward clinical success.
This workshop will gather experts to discuss:
- Understand the Current Delivery Landscape: Review leading approaches including GalNAc conjugation, LNPs, and novel carrier systems, and their applicability across different tissues
- Tackle Tissue-Specific Challenges: Explore practical solutions for crossing biological barriers such as the blood-brain barrier and targeting muscle, adipose tissue, or tumors
- Plan for Clinical Translation: Learn how to align delivery strategy with indication, safety requirements, and scalability considerations for late-stage development
Speakers:
